Long-term correction of murine phenylketonuria by viral gene transfer: liver versus muscle
Author:
Publisher
Wiley
Subject
Genetics (clinical),Genetics
Link
http://www.springerlink.com/index/pdf/10.1007/s10545-010-9044-3
Reference9 articles.
1. Ding Z, Georgiev P, Thöny B (2006) Administration-route and gender-independent long-term therapeutic correction of phenylketonuria (PKU) in a mouse model by recombinant adeno-associated virus 8 pseudotyped vector-mediated gene transfer. Gene Ther 13:587–593
2. Ding Z, Harding CO, Rebuffat A, Elzaouk L, Wolff JA, Thony B (2008) Correction of murine PKU following AAV-mediated intramuscular expression of a complete phenylalanine hydroxylating system. Molec Ther 16:673–681
3. Harding CO, Wild K, Chang D, Messing A, Wolff JA (1998) Metabolic engineering as therapy for inborn errors of metabolism—development of mice with phenylalanine hydroxylase expression in muscle. Gene Ther 5:677–683
4. Jung SC, Park JW, Oh HJ, Choi JO, Seo KI, Park ES, Park HY (2008) Protective effect of recombinant adeno-associated virus 2/8-mediated gene therapy from the maternal hyperphenylalaninemia in offsprings of a mouse model of phenylketonuria. J Korean Med Sci 23:877–883
5. Nagasaki Y, Matsubara Y, Takano H, Fujii K, Senoo M, Akanuma J et al (1999) Reversal of hypopigmentation in phenylketonuria mice by adenovirus-mediated gene transfer. Pediatr Res 45:465–473
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